Rigel Partners with CONNECT for Phase 2 Glioma Trial of Olutasidenib

Rigel Pharmaceuticals, Inc. has announced a new collaboration with CONNECT, a global network of pediatric cancer centers, to conduct a Phase 2 clinical trial. This trial aims to evaluate the combination therapy of REZLIDHIA® (olutasidenib) and temozolomide as a maintenance treatment for newly diagnosed pediatric and young adult patients with high-grade glioma (HGG) possessing an isocitrate dehydrogenase-1 (IDH1) mutation.

This collaboration involves integrating olutasidenib into CONNECT's TarGeT-D, a Phase 2 umbrella clinical trial aimed at HGG. Rigel will sponsor a specific arm of this trial where post-radiotherapy olutasidenib will be paired with temozolomide, before transitioning to olutasidenib monotherapy. This study will focus on pediatric and young adult patients under 39 years old with IDH1 mutation-positive HGG, including diffuse intrinsic pontine glioma (DIPG), a particularly aggressive brain tumor with few treatment options. Over four years, Rigel will fund up to $3 million and provide necessary study materials.

"We are thrilled to team up with CONNECT to investigate the potential of olutasidenib in high-grade glioma," said Raul Rodriguez, Rigel's President and CEO. He expressed confidence in olutasidenib's promise for various cancers involving the mIDH1 mutation and highlighted this trial as part of Rigel's strategy to expand its hematology-oncology pipeline.

The Phase 2 open label trial will be led by Drs. Santosh Valvi and Nicholas Gottardo from Perth Children's Hospital, Dr. Michael J Fisher from Children's Hospital of Philadelphia, and Dr. Maryam Fouladi from Nationwide Children's Hospital. The trial anticipates enrolling around 60 patients and aims to determine the progression-free survival rate. Additionally, it will assess the safety, tolerability, and pharmacokinetics of olutasidenib in the targeted patient population. Patient enrollment is expected to start in the first half of 2024.

Previously, a Phase 1b/2 trial involving 26 patients with relapsed or refractory (R/R) and predominantly enhancing gliomas with an IDH1 mutation demonstrated that olutasidenib, at a dose of 150 mg BID, was well tolerated. The study, published in Neuro-Oncology, also provided preliminary evidence of the drug's clinical activity and sustained disease control among a heavily pretreated cohort.

Rigel has ensured that REZLIDHIA is FDA-approved for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) who test positive for a susceptible IDH1 mutation.